KR20220019255A - 알파-1 항트립신 결핍증 (aatd)의 치료 방법 - Google Patents

알파-1 항트립신 결핍증 (aatd)의 치료 방법 Download PDF

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KR20220019255A
KR20220019255A KR1020227000043A KR20227000043A KR20220019255A KR 20220019255 A KR20220019255 A KR 20220019255A KR 1020227000043 A KR1020227000043 A KR 1020227000043A KR 20227000043 A KR20227000043 A KR 20227000043A KR 20220019255 A KR20220019255 A KR 20220019255A
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South Korea
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aat
drug substance
aat rnai
dose
pharmaceutical composition
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Pending
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KR1020227000043A
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Korean (ko)
Inventor
브루스 기븐
다운 크리스찬슨
제임스 해밀턴
젠 리
뤼 주
크리스틴 우드델
타오 페이
Original Assignee
애로우헤드 파마슈티컬스 인코포레이티드
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Publication of KR20220019255A publication Critical patent/KR20220019255A/ko
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
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    • C12N2310/3533—Halogen

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Wood Science & Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Zoology (AREA)
  • General Chemical & Material Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Dermatology (AREA)
  • Pulmonology (AREA)
  • Inorganic Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020227000043A 2019-06-06 2020-06-05 알파-1 항트립신 결핍증 (aatd)의 치료 방법 Pending KR20220019255A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962858059P 2019-06-06 2019-06-06
US62/858,059 2019-06-06
PCT/US2020/036359 WO2020247774A1 (en) 2019-06-06 2020-06-05 Methods for the treatment of alpha-1 antitrypsin deficiency (aatd)

Publications (1)

Publication Number Publication Date
KR20220019255A true KR20220019255A (ko) 2022-02-16

Family

ID=73652297

Family Applications (1)

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KR1020227000043A Pending KR20220019255A (ko) 2019-06-06 2020-06-05 알파-1 항트립신 결핍증 (aatd)의 치료 방법

Country Status (12)

Country Link
US (1) US20220305046A1 (enrdf_load_stackoverflow)
EP (1) EP3980540A4 (enrdf_load_stackoverflow)
JP (2) JP7697889B2 (enrdf_load_stackoverflow)
KR (1) KR20220019255A (enrdf_load_stackoverflow)
CN (1) CN114222820A (enrdf_load_stackoverflow)
AU (1) AU2020286462A1 (enrdf_load_stackoverflow)
BR (1) BR112021024534A2 (enrdf_load_stackoverflow)
CA (1) CA3141762A1 (enrdf_load_stackoverflow)
CO (1) CO2022000016A2 (enrdf_load_stackoverflow)
MX (1) MX2021015003A (enrdf_load_stackoverflow)
TW (1) TW202113081A (enrdf_load_stackoverflow)
WO (1) WO2020247774A1 (enrdf_load_stackoverflow)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW201620526A (zh) 2014-06-17 2016-06-16 愛羅海德研究公司 用於抑制α－１抗胰蛋白酶基因表現之組合物及方法
CN119082104A (zh) 2017-01-10 2024-12-06 箭头药业股份有限公司 α-1抗胰蛋白酶(AAT)RNAi物质、包含AAT RNAi物质的组合物和使用方法
EP4214316A2 (en) * 2020-09-15 2023-07-26 Arrowhead Pharmaceuticals, Inc. Methods for the reduction of z-aat protein levels
KR20240046843A (ko) * 2021-08-25 2024-04-11 노보 노르디스크 에이/에스 알파-1 항트립신 발현을 억제하기 위한 조성물 및 방법
AU2023330102A1 (en) * 2022-08-22 2025-02-27 Arrowhead Pharmaceuticals, Inc. Methods for the treatment of angptl3-related diseases and disorders

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NL9201440A (nl) 1992-08-11 1994-03-01 Univ Leiden Triantennaire clusterglycosiden, hun bereiding en toepassing.
US20050137153A1 (en) * 2002-02-20 2005-06-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of alpha-1 antitrypsin (AAT) gene expression using short interfering nucleic acid (siNA)
NZ554277A (en) * 2004-10-20 2010-09-30 Antisense Therapeutics Ltd Antisense modulation of integrin alpha 4 expression
TW201620526A (zh) * 2014-06-17 2016-06-16 愛羅海德研究公司 用於抑制α－１抗胰蛋白酶基因表現之組合物及方法
WO2017139616A1 (en) * 2016-02-10 2017-08-17 Arrowhead Pharmaceuticals, Inc. Methods of treatment for alpha-1 antitrypsin deficiency
EP3426261A4 (en) 2016-03-07 2020-03-25 Arrowhead Pharmaceuticals, Inc. Targeting ligands for therapeutic compounds
WO2017214518A1 (en) * 2016-06-10 2017-12-14 Alnylam Pharmaceuticals, Inc. COMPLETMENT COMPONENT C5 iRNA COMPOSTIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
JOP20170056B1 (ar) 2016-09-02 2021-08-17 Arrowhead Pharmaceuticals Inc مركبات ترابطية مستهدفة
CN119082104A (zh) * 2017-01-10 2024-12-06 箭头药业股份有限公司 α-1抗胰蛋白酶(AAT)RNAi物质、包含AAT RNAi物质的组合物和使用方法
SE540888C2 (en) * 2017-06-19 2018-12-11 Hepgene Medical Ab Novel nucleic acid molecules and their use in therapy

Also Published As

Publication number Publication date
JP7697889B2 (ja) 2025-06-24
EP3980540A4 (en) 2023-11-01
CN114222820A (zh) 2022-03-22
TW202113081A (zh) 2021-04-01
CA3141762A1 (en) 2020-12-10
MX2021015003A (es) 2022-01-24
WO2020247774A1 (en) 2020-12-10
JP2025096283A (ja) 2025-06-26
US20220305046A1 (en) 2022-09-29
CO2022000016A2 (es) 2022-04-08
JP2022535127A (ja) 2022-08-04
EP3980540A1 (en) 2022-04-13
BR112021024534A2 (pt) 2022-02-08
AU2020286462A1 (en) 2022-01-27

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Patent event date: 20220103

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Comment text: International Patent Application

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